NeurosciencebioRxivPreprint
Gene replacement therapy for Lafora disease in the Epm2a-/- mouse model
Luis Zafra‐Puerta, Daniel F. Burgos, Nerea Iglesias‐Cabeza…·José María Serratosa
This study uses gene replacement therapy in Epm2a-/- mice to treat Lafora disease, showing improvements in neurological symptoms and reduced Lafora body formation.
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